Policy Summit

Policy Summit Day Two: Trial Design, Regulatory Challenges, and Health Disparities

ASGCT Staff - September 24, 2020

On the second day of the Policy Summit, attendees began the morning with a keynote address from Peter Marks, M.D., Ph.D.

During the second day of the ASGCT Policy Summit, attendees heard from FDA, industry professionals and patient advocates about more of the regulatory issues, as well as legislative and access issues, in gene and cell therapies.

In a keynote address to begin the morning sessions, Peter Marks, M.D., Ph.D (FDA CBER), talked about the road from personalized medicine in the 1990s, which focused on finding the right drug off the shelf, to individualized medicine now—creating the right drug for the patient. Marks said the FDA needed a significant increase in staff to keep up with the workload of advancing gene therapies as quickly and safely as possible. COVID-19 has adversely affected all aspects of development as well.

Despite these challenges, “we are still extremely excited about gene therapy and advancing it as fast as we can,” Marks said.

He outlined the efforts led by the Foundation for the NIH, which FDA has been highly involved in, to increase standardization of AAV vectors, vector generation processes, and regulatory requirements, in order to be able to treat ultrarare diseases in a precompetitive space. This effort may be beneficial to the field in general, which will be able to leverage some of the ideas that emerge from it for streamlining development.

During the session on trial design considerations for gene therapy for rare diseases, Brad Glasscock, PharmD (BioMarin), emphasized that sponsors need greater regulatory predictability on factors such as the acceptance of surrogate endpoints. He noted that increasing the communication between sponsors and the Agency will be key. In his talk about patient-focused trial design, Daniel Chung, D.O. (Spark Therapeutics), showed data from a multi-luminance mobility test, which was the primary endpoint in the phase 3 trial for Luxturna. Seeing video of a patient going through the test really shows the value of the mobility endpoint, Chung said.

The next session focused on overcoming regulatory challenges in gene therapy trials. Attendees heard from industry and FDA speakers offering their perspectives on improving gene therapy development, selecting appropriate animal models for therapies, and addressing immunogenicity. Frequent and early communication and collaboration between sponsors and regulators became the common themes.

Anne-Virginie Eggimann (bluebird bio) said a life-cycle approach was important to CMC regulation for gene therapies to avoid delays, particularly for complex products. She said informal and proactive communication was key to making this happen. Ying Huang, Ph.D. (FDA), agreed that early communication was important. INitial Targeted Engagement for Regulatory Advice on CBER producTs (INTERACT) meetings would help developers get early feedback on potential therapies, she said.

The afternoon sessions began with keynote speaker Michelle McMurry-Heath, M.D., Ph.D. (BIO), who discussed the urgent need of the scientific community to address treatment and coverage disparities that make it more difficult for vulnerable communities to access gene and cell therapies.

“Access to science is the social justice issue of our age,” she said, adding that the pandemic has laid bare the links between race and poverty in the U.S. and around the world.

McMurry-Heath acknowledged the pressure that many scientists are feeling, but called on attendees to double down on scientific integrity. Speaking about COVID-19 vaccine efforts, she said we can’t weaken the public trust by allowing an unproven vaccine to be made available. We must not let speed overcome rationale and common sense.

The afternoon continued with a session about evidence generation for regulators and payers, which included Eric Faulkner (Evidera), Americo Cicchetti (Universitá Cattolica del Sacro Cuor), and Jeet Guram, M.D. (FDA). Faulkner gave an overview of evidence generation for regulators and payers, Cicchetti provided a perspective on evidence requirements and related policies in Italy, and Guram shared his knowledge from his current role in the FDA and his recent role within CMS. Both Faulkner and Guram encouraged evaluating outcomes that are easier to track to better facilitate value-based payment arrangements. Guram indicated that the spirit of the CMS proposed rule released in June was to propose new regulatory flexibilities (multiple Medicaid best prices) to value-based payment models, and that CMS has been thinking a lot about payment over time, which is a discussion that will continue. Cicchetti noted that in Italy there has been discussion about creating an extra fund dedicated to reimbursement of gene and cell therapies.

The final session of the day focused on sickle cell disease and offered researcher, patient, and developer perspectives on treatments. John Tisdale, M.D. (NHLBI) gave an overview of the disease, which affects about 100,000 patients in the U.S., with 85 to 90% being African Americans. He said there are many barriers to care: lack of primary physician training, patients not seeing hematology specialists often enough, lack of available centers in many states, patient concentration in poor or rural communities, and a lack of patient knowledge and trust in the medical community. Beverley Francis-Gibson (Sickle Cell Disease Association of America) said education is key; the more patients know and understand, the more they can be involved in treatment options. Samarth Kulkarni, Ph.D. (CRISPR Therapeutics) and Nick Leschly (bluebird bio) both discussed treatments that their companies are working on. CTX001 is a gene editing approach currently in clinical trials, Kulkarni said, and aims to treat sickle cell by elevating fetal hemoglobin. As of June 2020, the treatment was successful for Victoria Gray, the first patient with a genetic disorder to get treated in the U.S. with CRISPR. Leschly showed two years of data from 14 patients who had a 99.5% reduction in the annualized rate of VOCs after undergoing an investigational gene therapy treatment.

During the panel discussion, speakers agreed that access to clinical trials and to gene therapies upon approval will be a huge issue for patients and everyone in the field has a role to play in demystifying the science of gene therapy and solving the patient access and payment policy challenges ahead.

“The science is here, the medicine is here,” Leschly said. “The rest is up to us.”

ASGCT recently released resources on sickle cell disease that you can view on the Patient Education site. If you missed anything at the Policy Summit, all sessions are free for registered attendees to watch on the meeting website for 30 days. You can read our recap of the first day on our blog.

Join us tomorrow for the final day of the 2020 Policy Summit!


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